Key Takeaways
Theriva Biologics Inc. (NYSE: TOVX) saw its shares gain after announcing it reached an alignment with the U.S. Food and Drug Administration on the design of a pivotal Phase 3 trial for its lead cancer drug, VCN-01, in patients with metastatic pancreatic ductal adenocarcinoma (PDAC). The agreement marks a critical step in de-risking the regulatory path for a potential new therapy in a notoriously difficult-to-treat cancer.
“The first quarter of 2026 was marked by encouraging regulatory progress,” said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics, in a May 5 statement. “We were pleased to receive the minutes from our end-of-Phase 2 meeting with the FDA and align on the major elements of our proposed pivotal Phase 3 trial.”
The company reported cash and cash equivalents of $14.4 million as of March 31, 2026, providing a cash runway into the first quarter of 2027. The announcement comes after its prior VIRAGE Phase 2b trial met its primary endpoints, demonstrating improved overall survival (OS) and progression-free survival (PFS) in patients who received VCN-01 with standard chemotherapy compared to chemotherapy alone.
The alignment with the FDA provides a clearer path forward for VCN-01, a systemically administered oncolytic adenovirus designed to selectively replicate within tumor cells. For investors, the agency's feedback reduces uncertainty around the final-stage study required for a potential Biologic Licensing Application (BLA), a major hurdle for any clinical-stage biotech company. Pancreatic cancer has one of the lowest survival rates among all cancers, with only 13% of patients surviving five years, creating a high unmet need for new treatments.
The upcoming Phase 3 trial design will incorporate repeated "macrocycles" of VCN-01 dosing alongside standard-of-care chemotherapy. This approach is based on findings from the VIRAGE study, which observed greater improvements in OS and PFS in patients who received two doses of VCN-01.
VCN-01 is an oncolytic adenovirus engineered to break down the dense stromal barrier of tumors, a key defense mechanism for pancreatic cancer, while promoting an anti-tumor immune response. To refine the new dosing strategy, Theriva plans to launch a small dosing feasibility study at a single site in Spain in the second half of 2026.
Theriva's reported cash balance of $14.4 million is central to its ability to execute on the upcoming trials. The company's first-quarter net loss was $2.0 million, with research and development expenses falling 88% to $355,000 following the completion of the VIRAGE trial.
Beyond pancreatic cancer, Theriva is also developing VCN-01 for retinoblastoma, a rare eye cancer in children. The company has made the drug available under a compassionate use program and has treated two patients. It is currently designing a potential Phase 2/3 trial for this indication, further diversifying its clinical pipeline.
Theriva’s progress comes amid a surge of renewed hope in the pancreatic cancer field. Just weeks ago, the FDA granted expanded access to daraxonrasib, an experimental drug from Revolution Medicines that targets KRAS mutations, which are found in over 90% of pancreatic cancers. In a pivotal trial, daraxonrasib nearly doubled median overall survival to 13.2 months.
While VCN-01 uses a different mechanism, the parallel progress highlights a potential "paradigm shift" in treating the disease, as described by Dr. Suneel Kamath of the Cleveland Clinic to Healio. The success of targeted agents like daraxonrasib and novel approaches like Theriva's oncolytic virus suggest that after decades of limited progress, a new era of more effective therapies may finally be arriving for patients.
This article is for informational purposes only and does not constitute investment advice.
